RGX-202 in Duchenne muscular dystrophy on track for topline results 1H 2026 and BLA submission mid-2026 Pivotal trial enrollment accelerated, expected to complete in October 2025 ahead of previous guidance Clemidsogene lanparvovec (RGX-121) on track to be first gene therapy and one-time...
Related Questions
How does the anticipated BLA submission timeline in mid‑2026 compare to competitors' gene therapy programs in Duchenne muscular dystrophy?
What impact might the progress of Clemidsogene lanparvovec (RGX‑121) have on the valuation of the broader RGX pipeline?
How does the current financial performance (e.g., net loss, R&D spend) compare to peers in the gene‑therapy space?
What are the potential market size and pricing implications for RGX-202 if it achieves topline results in H1 2026?
What are the potential partnership or licensing opportunities that could arise from the successful development of RGX‑202 and RGX‑121?
How might the updated guidance affect analyst consensus estimates and target prices for the stock?
What regulatory risks remain for the BLA submission, and how could they affect the stock’s volatility?
How will the accelerated enrollment and earlier completion of the RGX-202 pivotal trial affect the company's projected revenue and cash flow forecasts?
Will the earlier trial completion influence the company's burn rate and runway, potentially affecting dilution or financing needs?
How might the market react to the company's ability to meet or exceed the accelerated timelines versus its previous guidance?